Patients With CLL Are Willing to Trade Efficacy for Less Severe Side Effects

Katie Kosko

PATIENTS WITH CHRONIC LYMPHOCYTIC leukemia (CLL) are willing to accept a shorter duration of progression-free survival (PFS) if it means avoiding serious side effects from treatment, according to the results of a study published online in Blood Advances, a journal of the American Society of Hematology.

Researchers from across the country also found that out-of-pocket costs played a deciding factor, too.

“People have preferences based on what they value the most,” Carol Mansfield, Ph.D., senior research economist at RTI Health Solutions, said in an interview with CURE®. “As medicine moves toward a more patient-involved paradigm, we are learning that people have different preferences and circumstances, so there may not be a ‘one size fits all’ medicine choice.”

The Leukemia & Lymphoma Society sent an email survey to more than 4,000 patients with CLL, 384 of whom consented to and completed it. Patients were, on average, aged 65 years; approximately half were men, and most were white.

The participants were asked to choose between hypothetical treatment options, each defined by five variable attributes — PFS, mode of administration, typical severity of diarrhea, chance of serious infection and chance of organ damage.

Although patients placed the highest importance on longer PFS, reductions in the risk of side effects also appeared to be important. For example, patients said they would need, on average, an additional 36 months of PFS in order to accept an increased risk of serious infection. In turn, that amount of added efficacy would offset a change in side effects — in this case, patients going from having no risk to a 30 percent chance of an adverse event, according to the study authors. “Patients value both efficacy and quality of life during treatment, and they are often willing to make trade-offs,” Mansfield said.

In addition, cost of care plays a significant role in treatment decision making. When cost was not a factor, 91 percent of respondents said they would choose the medicine with the longest PFS. But when the choice involved two medicines with out-of-pocket costs, patients opted for the cheaper medicine despite the lower PFS.

In a scenario with a $400 difference between out-of-pocket costs, 75 percent of patients chose the cheaper medicine. In the case with a $75 difference per month, half (50 percent) chose the lower-cost medicine.

“Cost is complicated. It varies from patient to patient, based on insurance and access to other sources of financial aid,” Mansfield said. “The reality is that the costs we presented in the survey were high enough that some patients couldn’t afford them.”

The study authors hope that these findings will encourage health care providers to have frank and open discussions with patients about how different treatments will affect their lives, Mansfield said.

Having providers help manage side effects can make a big difference and may even convince patients to stay on drugs that have higher efficacy and worse side effects, she added. “Choosing a treatment is more than a go or no-go decision,” Mansfield said. “Patients don’t always understand the trade-offs involved with different treatment plans. Once they understand the impact of their choices, they can make decisions based on what they are willing to tolerate and what they are willing to risk.”